Patients want new treatments to improve their quality of life. However, do the results reported by the typical patient adequately reflect the effect of the new treatment on the factors that patients care about most? paper from Pervito et al. (2022) He argues that the answer is “no.”
The article notes that patients not only want an improved functional state, but also fewer days in the hospital and avoid situations where there is an “inability to plan” and disruptions at work/school. Furthermore, the other suggests the development of patient-centered primary effect groups (PC-CIS). What is PC-CIS?
…Patient Centered Primary Impact Groups (PC-CIS) [are] A patient-derived and prioritized list of the effects of the disease and/or its treatments on the patient (and/or their family and caregivers). The term ‘effects’ is broad and intentionally inclusive, including short and long-term health outcomes and any other relevant effects (eg, caregiver/family stress, economic burden, job loss).
The authors grouped the effects into disease-related effects (eg, quality of life, employment status), treatment-related effects (eg, adverse events, risk of failure), and financial impact (eg, occupational impact, insurance coverage). and influence on family/caregivers (eg, stress on family, commitment to family time). There have been a number of Core Outcome Sets (COS) developed; Indeed, the University of Liverpool’s primary outcome measures in efficacy trials (CometThe initiative includes the published COS database. To move from COS to priority PC-CIS, the article cites the National Health Council (NHC) framework for PC-CIS development (shown in the figure below).
There are a number of challenges to implementing PC-CIS. These include (1) PC-CIS may need to be developed per disease, which is a challenge due to the volume of diseases, (2) PC-CIS per disease may vary by country as well, (3) there is no standardized process for PC-CIS development, and ( 4) It is not clear which organization(s) should be responsible for developing PC-CIS. Despite these challenges, properly measuring how treatments affect the outcomes that patients care about most is a commendable goal.
